Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities.
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| Title: | Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities. |
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| Authors: | Cheff DM; National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA.; Division of Biochemistry, Department of Medical Biochemistry and Biophysics, Karolinska Institutet, 171 77, Stockholm, Sweden., Muotri AR; Department of Pediatrics, University of California, San Diego, San Diego, CA, USA.; Department of Cellular and Molecular Medicine, University of California, San Diego, San Diego, CA, USA., Stockwell BR; Department of Biological Sciences, Columbia University, New York, NY, USA.; Department of Chemistry, Columbia University, New York, NY, USA., Schmidt EE; Department of Microbiology and Immunology, Montana State University, Bozeman, MT, USA., Ran Q; Department of Cell Systems and Anatomy, University of Texas Health Science Center, San Antonio, San Antonio, TX, USA.; Research and Development Service, South Texas Veterans Health Care System, San Antonio, TX, USA., Kartha RV; Department of Experimental and Clinical Pharmacology, Center for Orphan Drug Research, College of Pharmacy, University of Minnesota, Minneapolis, MN, USA., Johnson SC; Department of Neurology, University of Washington, Seattle, WA, USA.; Department of Anesthesiology and Pain Medicine, University of Washington, Seattle, WA, USA.; Center for Integrative Brain Research, Seattle Children's Research Institute, Seattle, WA, USA., Mittal P; In-Depth Genomics, Bellevue, WA, USA., Arnér ESJ; Division of Biochemistry, Department of Medical Biochemistry and Biophysics, Karolinska Institutet, 171 77, Stockholm, Sweden.; Department of Selenoprotein Research, National Institute of Oncology, Budapest, 1521, Hungary., Wigby KM; Department of Pediatrics, Division of Genetics, San Diego and Rady Children's Hospital-San Diego, University of California, San Diego, CA, USA.; Rady Children's Institute for Genomic Medicine, San Diego, CA, USA., Hall MD; National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA., Ramesh SK; CureGPX4.org, Seattle, WA, USA. sanath@GPX4.org. |
| Source: | Orphanet journal of rare diseases [Orphanet J Rare Dis] 2021 Oct 23; Vol. 16 (1), pp. 446. Date of Electronic Publication: 2021 Oct 23. |
| Publication Type: | Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't; Review |
| Journal Info: | Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1750-1172 |
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| DOI: | 10.1186/s13023-021-02048-0 |
